Company |
Product |
Description |
Indication |
Status |
Date |
CANCER | |||||
Amgen Inc. (Thousand Oaks, Calif.) |
Blinatumomab |
Bispecific antibody B-precursor |
Newly diagnosed negative B-lineage acute lymphoblastic leukemia |
Launched a randomized phase III trial comparing overall survival after treatment with blinatumomab in combination with chemotherapy vs. chemotherapy alone in adults ages 35 to 70 years |
1/13/14 |
Celator Pharmaceuticals Inc. (Ewing, N.J.) |
CPX-351 |
Cytarabine: daunorubicin; liposome injection |
High-risk acute myeloid leukemia |
Phase III data of CPX-351 vs. the conventional cytarabine and daunorubicin treatment regimen as a first-line therapy in older patients has reached 50% of the study’s planned enrollment of 300 patients |
1/10/14 |
Cell Therapeutics Inc. (Seattle) |
Opaxio |
Paclitaxel poliglumex |
Ovarian cancer |
Completed patient enrollment in the GOG-0212 phase III trial testing Opaxio as a maintenance therapy; the primary endpoint is overall survival, while secondary endpoints include progression-free survival, safety and quality of life |
1/29/14 |
Celsion Corp. (Lawrenceville, N.J.) |
Thermodox |
Heat-activated liposomal encapsulation of doxorubicin |
Primary liver cancer |
Phase III HEAT study data supported the continued clinical development in a prospective phase III pivotal study; although HEAT did not meet its primary endpoint of progression-free survival, in the patient subgroup treated in the Thermodox arm whose RFA procedures lasted longer than 45 minutes, clinical results indicated a 55% improvement in overall survival (p = 0.0495) |
1/28/14 |
Immunomedics Inc. (Morris Plains, N.J.) |
90Y |
Yttrium-90-clivatuzumab tetraxetan |
Pancreatic cancer |
Launched a phase III registration study |
1/13/14 |
Medivation Inc. (San Francisco) and Astellas Pharma Inc. (Tokyo) |
Xtandi |
Enzalutamide; oral androgen receptor inhibitor |
Post-chemotherapy |
Phase III data showed a statistically significant overall survival benefit compared with placebo, reducing the risk of death by 29% (HR = 0.71; p < 0.0001), compared with placebo, and the benefit held despite the use of subsequent therapies, at a rate of 40% in the Xtandi arm, 70% in placebo |
1/30/14 |
Patrys Ltd. (Melbourne, Australia) |
PAT-SC1 |
The target antigen, CD55, is an isoform of the membrane-bound CD55 |
Gastric cancer |
Data from a 10 year follow-up study showed that survival is significantly higher for PAT-SC1 treated patients as compared to an historical control group of patients who underwent a gastric resection but were not treated with PAT-SC1, which was well tolerated in all treated patients with no significant side effects |
1/23/14 |
Pharmacyclics Inc. (Sunnyvale, Calif.) and Janssen Biotech Inc. (unit of Johnson & Johnson; New Brunswick, N.J.) |
Imbruvica |
Ibrutinib |
Relapsed or refractory chronic lymphocytic leukemia or small lymphocytic |
An independent data monitoring committee unanimously recommended the phase III study of Imbruvica vs. Arzerra be stopped early because the primary endpoint and a key secondary endpoint have been met |
1/8/14 |
CARDIOVASCULAR | |||||
Amgen Inc. (Thousand Oaks, Calif.) |
Evolocumab |
Anti-PCSK9 antibody |
High cholesterol |
Phase III data in patients with high cholesterol who cannot tolerate statins met its co-primary endpoints |
1/24/14 |
Cardium Therapeutics Inc. (San Diego) |
Generx |
Alferminogene tadenovec, Ad5FGF-4 |
Myocardial ischemia due to arteriosclerosis |
Phase III data suggest that a one-time non-surgical administration of Generx can boost cardiac blood flow (myocardial perfusion) |
1/2/14 |
Emmaus Medical Inc. (Torrance, Calif.) |
L-glutamine |
Levoglutamide |
Sickle cell disease |
Patient visits were completed in the phase III trial |
1/9/14 |
Oxygen Biotherapeutics Inc. (Morrisville, N.C.) |
Levosimendan |
Calcium sensitizer |
Cardiac disease |
Company is moving the drug into a phase III study under an SPA; it will enroll about 760 cardiac patients |
1/7/2014 |
CENTRAL NERVOUS SYSTEM | |||||
Endo Pharmaceuticals Inc. (Malvern, Pa.; subsidiary of Endo Health Solutions Inc.) and Biodelivery Sciences International Inc. (Raleigh, N.C.) |
BEMA |
Buprenorphine |
Chronic pain |
Top-line results from the pivotal phase III study showed it met its primary efficacy endpoint, showing that BEMA buprenorphine resulted in improved chronic pain relief (p < 0.005) compared to placebo |
1/27/14 |
Envivo Pharmaceuticals Inc. (Watertown, Mass.) |
Encenicline-hydrochloride |
Alpha-7 potentiator |
Alzheimer's disease |
Launched a second phase III program |
1/23/14 |
GASTROINTESTINAL | |||||
NPS Pharmaceuticals Inc. (Bedminster, N.J.) |
Gattex |
Teduglutide (rDNA origin) |
Short bowel syndrome |
Started a global registration study in pediatric patients who are dependent on parenteral support |
1/3/14 |
INFECTION | |||||
Melinta Therapeutics Inc. (New Haven, Conn.) |
Delafloxacin |
A fluoroquinolone |
Uncomplicated gonorrhea |
Began a phase III trial |
1/10/14 |
Paxvax Inc. (Menlo Park, Calif.) |
PXVX0200 |
Also known as CVD 103-HgR; oral vaccine |
Cholera |
Interim phase III data showed the vaccine appeared well tolerated and the rate of diarrhea was reduced in the vaccine group compared with placebo |
1/10/14 |
INFLAMMATORY | |||||
Ampio Pharmaceuticals Inc. (Greenwood, Colo.) |
Ampion |
Aspartyl-alanyl diketopiperazine |
Osteoarthritis of the knee |
Received institutional review board approval and FDA investigational new drug application clearance, and patient enrollment has commenced for a phase III trial |
1/14/14 |
Shield Therapeutics Ltd. (London) |
ST10 |
An oral ferric iron replacement therapy |
Anemia caused by inflammatory bowel disease |
Pivotal phase III data showed the product could replace the need to administer intravenous iron; patients receiving the therapy had a mean increase in hemaglobin levels of 2.3 grams/deciliter at 12 weeks, meeting the primary endpoint |
1/7/14 |
MISCELLANEOUS | |||||
Debiopharm Group (Lausanne, Switzerland) |
Triptorelin pamoate |
A 22.5 mg, six-month formulation |
Central precocious puberty |
Completed the recruitment of patients for a phase III study |
1/14/14 |
Mesoblast Ltd. (Melbourne, Australia) |
MPCs |
Allogeneic mesenchymal precursor cells |
Chronic moderate to severe discogenic low-back pain |
Phase III data showed a single injection into degenerating intervertebral discs reduced low-back pain and improved function for at least 12 months |
1/31/14 |
Nymox Pharmaceutical Corp. (Hasbrouck Heights, N.J.) |
NX-1207 |
BPH candidate |
Benign prostatic hyperplasia |
Two pivotal studies – NX02-0017 and NX02-0018 – have completed enrollment |
1/29/14 |
Prosensa Holding N.V. (Leiden, the Netherlands) |
Drisapersen |
An antisense oligonucleotide which induces exon skipping of exon 51 |
Duchenne muscular dystrophy |
Phase III data showed a 49 m difference between those on continual treatment (n = 52) and those who had been on placebo for 48 weeks followed by active drug (n = 31); those previously participating in the DMD114117 study (DEMAND II) showed a 52 m difference at 96 weeks |
1/17/14 |
Relypsa Inc. (Redwood City, Calif.) |
Patiromer |
An oral potassium binder |
Hyperkalemia |
Results from the pre-defined exploratory endpoints from its two-part pivotal phase III trial show that as a result of recurrent hyperkalemia significantly more placebo patients required dose modification of their RAASi therapies (62%) than patiromer patients (6%); with more patiromer patients (94%) still on RAASi medication at the end of the trial than placebo patients (48%) |
1/14/14 |
Repros Therapeutics Inc. (The Woodlands, Texas) |
Androxal |
Enclomiphene |
Testosterone replacement therapy |
Initiated two clinical studies comparing Androxal to the leading U.S. testosterone replacement therapy; each study will enroll 120 men, 40 each into three parallel arms: Androxal, approved topical gel and placebo |
1/9/14 |
ZS Pharma Inc. (Coppell, Texas) |
ZS-9 |
A highly selective oral sorbent designed specifically to trap potassium ions |
Hyperkalemia |
Reported top-line results from ZS003, its pivotal phase III trial, showing the drug met the pre-specified endpoint for the 12-day extended treatment phase, demonstrating that both 5 g and 10 g of ZS-9 given once-daily maintained a statistically and clinically significant reduction in serum potassium (K+) relative to placebo; it also was well tolerated with a gastrointestinal (GI) adverse event rate and an overall adverse event rate similar to placebo across all doses tested |
1/14/14 |
Notes BLA = Biologics license application; CMA = Continuous marketing application; FDA = Food and Drug Administration; IND = Investigational new drug application; NDA = New drug application; PDUFA = Prescription Drug User Fee Act; SPA = Special protocol assessment. Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. The date indicated refers to the BioWorld Today issue in which the news item can be found. |